Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis
Published 2011 View Full Article
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Title
Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis
Authors
Keywords
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Journal
HUMAN MOLECULAR GENETICS
Volume 20, Issue 23, Pages 4569-4581
Publisher
Oxford University Press (OUP)
Online
2011-09-01
DOI
10.1093/hmg/ddr391
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- The Leber congenital amaurosis protein, AIPL1, is needed for the viability and functioning of cone photoreceptor cells
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- Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors
- (2009) Mei Hong Tan et al. HUMAN MOLECULAR GENETICS
- AIPL1, a Protein Associated with Childhood Blindness, Interacts with α-Subunit of Rod Phosphodiesterase (PDE6) and Is Essential for Its Proper Assembly
- (2009) Saravanan Kolandaivelu et al. JOURNAL OF BIOLOGICAL CHEMISTRY
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- Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial
- (2008) William W. Hauswirth et al. HUMAN GENE THERAPY
- Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer
- (2008) Jeannette Bennicelli et al. MOLECULAR THERAPY
- Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
- (2008) Albert M. Maguire et al. NEW ENGLAND JOURNAL OF MEDICINE
- Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis
- (2008) James W.B. Bainbridge et al. NEW ENGLAND JOURNAL OF MEDICINE
- Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses
- (2008) L. Zhong et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
- (2008) A. V. Cideciyan et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
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